On November 4, 2019 Unum Therapeutics Inc. (NASDAQ: UMRX), a clinical-stage biopharmaceutical company focused on developing curative cell therapies for cancer, reported a strategic shift to focus development on its ACTR and BOXR product candidates in solid tumors and supportive platform capabilities (Press release, Unum Therapeutics, NOV 4, 2019, View Source [SID1234550246]).

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"We are uniquely positioned to address the challenge of treating solid tumor cancers, and now is the time to focus our efforts, having recently validated our ACTR technology in the hematologic setting and with preclinical data emerging from BOXR1030, the first product candidate from our BOXR platform. Our ACTR technology enables selective T cell targeting for on-tumor attack, while our BOXR platform makes it possible to overcome solid tumor immunosuppression, the fundamental challenge that has limited the effectiveness of cell therapies," said Chuck Wilson Ph.D., President and Chief Executive Officer of Unum. "Our priorities in solid tumors include completing the ongoing Phase 1 trial of ACTR707 in HER2+ cancers; advancing BOXR1030 towards the clinic with an anticipated IND filing in 2020; and expanding our BOXR platform to accelerate discovery of new product candidates across a broad range of immune cell therapies, including both autologous and allogeneic approaches."

ACTR707 was engineered for properties that optimize its function in solid tumors including increased proliferation, cytokine secretion, and persistence. With Unum’s focus on developing therapies for solid tumors, the company will de-prioritize investment in its hematologic programs. Testing through the first four dose levels in the ongoing ATTCK-20-03 Phase 1 trial in non-Hodgkin lymphoma has now established proof-of-concept for ACTR707. Given favorable tolerability to date at relatively low doses, Unum is announcing today plans to continue limited dose escalation to inform potential future development of the program in 2020.

Separately, Unum and its partner, Seattle Genetics, Inc., have suspended further dose-escalation of the ATTCK-17-01 Phase 1 trial of ACTR087 with SEA-BCMA in multiple myeloma pending a further review of this program. No dose-limiting toxicities (DLTs) following ACTR087 administration were reported and no severe adverse events of cytokine release syndrome (CRS) or neurologic events have been observed to date.

Solid Tumor Program Highlights

Phase 1 ATTCK-34-01 Trial: ACTR707 combined with trastuzumab to treat advanced HER2+ solid tumor cancers. Five clinical sites are now activated to support the Phase 1 trial that is currently enrolling patients. Unum expects to report preliminary safety data from patients treated in the first dose cohort of the trial by the end of this year and to report safety and clinical response data from multiple dose cohorts in 2020.

BOXR1030: Incorporating the GOT2 transgene and targeting GPC3+ solid tumor cancers. Unum’s first product candidate selected from its Bolt-On Chimeric Receptor (BOXR) platform, BOXR1030, continues to progress towards first-in-human clinical trials. BOXR1030 expresses a glypican-3 (GPC3) targeted chimeric antigen receptor (CAR) and leverages the "bolt-on" transgene glutamic-oxaloacetic transaminase 2 (GOT2) to improve T cell function in the solid tumor microenvironment by enhancing T cell metabolism. Preclinical studies have characterized the mechanism of action of BOXR1030’s bolt-on transgene with further details to be presented at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) conference during November 6-10, 2019. Based on recent progress, Unum now plans to file an investigational new drug (IND) application for BOXR1030 in late 2020, enabling subsequent clinical testing in GPC3+ cancers.

BOXR Platform Expansion: Unum’s BOXR platform was established over two years ago with the aim of discovering novel transgenes that can be co-expressed with chimeric-targeting receptors to improve the function of T cells in the solid tumor microenvironment. As part of its strategic shift to target solid tumors, Unum will be scaling up its BOXR platform capabilities with the objectives of: (1) expanding the scope of biological mechanisms and transgenes in its proprietary BOXR library, (2) enabling BOXR bolt-on applications for a broad range of immune cell therapies, including both autologous and allogeneic approaches, and (3) advancing new BOXR product candidates into the clinic.
Hematologic Program Highlights

Phase 1 ATTCK-20-03 trial: ACTR707 combined with rituximab for relapsed/refractory non-Hodgkin lymphoma. As a preliminary update provided today for the six patients treated in Cohort 4 (80M ACTR707+ T cells), complete response was achieved at the first response assessment in two of six patients as of the October 2019 analysis, yielding a complete response rate of 40% (eight of 20 patients) in Cohorts 1 through 4. Of the eight complete responders, four remained in complete response at six months of follow-up, two remain in complete response but have not yet reached the six-month timepoint for evaluation, and two progressed before the six-month timepoint. In Cohorts 1 through 4, ACTR707 was well-tolerated in combination with rituximab. No DLTs, no adverse events of CRS, and no severe neurological adverse events including neurotoxicity have been reported as of the October 2019 cutoff. Further results will be presented at the American Society for Hematology (ASH) (Free ASH Whitepaper) Annual Meeting during December 7-10, 2019. Unum plans to enroll up to two additional cohorts (three to four patients per cohort) in the trial, escalating the maximum dose up to 180M ACTR707+ T cells. With patient screening and planned dosing underway, Unum plans to report preliminary results from this dose escalation during 2020. The ability to differentiate on both efficacy and safety relative to currently available therapies and those in development from these additional cohorts will drive a decision during 2020 whether to advance the program into an expanded dose cohort and potential pivotal studies.

Phase 1 ATTCK-17-01 trial: ACTR087 combined with SEA-BCMA for relapsed/refractory multiple myeloma. Two additional cohorts of patients have been treated in the Phase 1 trial in 2019, escalating doses of the SEA-BCMA antibody to 2.0 mg/kg and of the ACTR087+ T cells to 50M. Unum and Seattle Genetics have suspended further dose-escalation of the trial and are reviewing the next steps with this program.
Investor Call and Webcast Information

Unum will host a live conference call and webcast today, November 4, 2019, at 4:30 p.m. ET, to discuss these company updates. To access the call, please dial 866-300-3411 (domestic) or 636-812-6658 (international) and refer to conference ID number 2177408. A webcast will be available at unumrx.com at least 10 minutes before the event begins. The archived webcast will be available at the same location approximately two hours after the event and will be archived for 90 days.

On November 4, 2019 Epizyme, Inc. (Nasdaq: EPZM), a late-stage biopharmaceutical company developing novel epigenetic therapies, reported funding agreements with Royalty Pharma and its affiliate Pharmakon Advisors that, in aggregate, could bring in up to $270 million in capital, significantly strengthening Epizyme’s financial position and extending its operating runway into at least 2022 (Press release, Epizyme, NOV 4, 2019, View Source [SID1234550263]).

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"These funding transactions support our strategy of becoming a fully integrated, commercial biopharmaceutical company, and provide us significant optionality for the future," said Robert Bazemore, president and chief executive officer of Epizyme. "We are well capitalized to fully fund the anticipated launches of tazemetostat for epithelioid sarcoma and follicular lymphoma next year, and further invest in both the continued development of tazemetostat and our epigenetic pipeline. Royalty Pharma has an outstanding reputation for investing in promising, late-stage companies, and we are thrilled to have their support as we head into the next phase of our evolution."

As part of the agreements, Royalty Pharma will make an upfront payment of $100 million for shares of Epizyme common stock based on a price of $15 per share, representing a 27% premium to the prior trading day’s closing price. Epizyme also has an 18-month option to sell an additional $50 million of its common stock to Royalty Pharma at then prevailing prices, not to exceed $20 per share, and Royalty Pharma has a three-year option to purchase an additional 2.5 million shares of Epizyme common stock at $20 per share.

Epizyme and Royalty Pharma have also agreed to reduce the existing royalty rates owed by Epizyme to Royalty Pharma on worldwide sales of tazemetostat outside Japan at specified annual net sales levels. This reduction follows Royalty Pharma’s acquisition of certain future royalty streams on sales of tazemetostat in all regions except for Japan, which Epizyme previously owed to Eisai as part of their April 2015 amended and restated agreement. In addition, Epizyme has assigned to Royalty Pharma the future royalty streams on tazemetostat sales in Japan previously owed to Epizyme by Eisai.

"We are very pleased to partner with Epizyme, particularly given the tremendous progress in advancing tazemetostat over the course of 2019," stated Pablo Legorreta, founder and chief executive of Royalty Pharma. "With impressive data in treating both solid tumors and hematologic malignancies, we share the company’s excitement for tazemetostat’s broad therapeutic potential and are committed to supporting tazemetostat’s further development and anticipated launches to fully maximize its commercial opportunity. We look forward to working closely with this talented team as they transition into a commercial-stage organization."

As part of the transactions, Mr. Legorreta will be appointed to Epizyme’s Board of Directors.

Under a separate agreement, Epizyme has established a $70 million loan facility with Pharmakon Advisors, an affiliate of Royalty Pharma, which will fund the regulatory milestones owed to Eisai for the NDA submissions and U.S. approvals for tazemetostat for epithelioid sarcoma and follicular lymphoma. The facility may be drawn down in up to three tranches in conjunction with achievement of the milestones and is expandable by up to $300 million following approval of tazemetostat in follicular lymphoma, subject to mutual agreement.

For a further description of terms of the transaction agreements, please refer to Epizyme’s Form 8-K filed today.

Wilmer Cutler Pickering Hale and Dorr LLP acted as legal advisor to Epizyme; Goodwin Procter LLP, Dechert LLP and Maiwald Patentanwalts- und Rechtsanwalts GmbH acted as legal advisors to Royalty Pharma; and, Akin Gump Strauss Hauer & Feld LLP acted as legal advisor to Pharmakon Advisors.

On November 4, 2019 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer and other indications, reported that it has entered into a securities purchase agreement with existing and new institutional investors to raise $32 million (Press release, Pieris Pharmaceuticals, NOV 4, 2019, View Source [SID1234550211]).

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The private placement was led by BVF Partners L.P., with significant additional participation from EcoR1 Capital, Aquilo Capital Management, Surveyor Capital (a Citadel company), and Samsara BioCapital.

The private placement will consist of 9,014,960 units, at a price of $3.55 per unit. Each unit will consist of (i) one share of Pieris’ common stock or 0.001 non-voting Series C convertible preferred stock, and (ii) one immediately-exercisable warrant to purchase one share of common stock at an exercise price of $7.10 per share. Each share of non-voting Series C convertible preferred stock is convertible into 1,000 shares of Pieris common stock, provided that conversion will be prohibited if, as a result, the holder and its affiliates would own more than 9.99% of the total number of shares of Pieris common stock then outstanding.

The warrants are intended to facilitate Pieris’ exercise of its co-development option for PRS-060/AZ1402 following the conclusion of a positive phase 2a study. If top-line results of that study disclose achievement of the primary efficacy endpoint and the stock reaches a pre-specified price, then the warrants will expire sixty days following such disclosure and may only be exercised for cash. Otherwise, the warrants will be exercisable for a period of five years from the date of issuance.

The closing of the financing is expected to take place on or about November 6, 2019 and is subject to standard closing conditions. Pieris expects to use the proceeds from the financing for continued development of its immuno-oncology franchise, including PRS-343, its 4-1BB/HER2 bispecific, and PRS-344, its 4-1BB/PD-L1 bispecific in co-development with Servier. The Company also expects to use these proceeds for advancement of its proprietary pipeline of inhalable respiratory drug candidates following proof-of-mechanism with respect to PRS-060/AZ1402, validating the inhaled Anticalin platform as a potential drug class, as well as for working capital and general corporate purposes.

William Blair & Company, L.L.C. acted as sole placement agent for the transaction.

The securities to be sold in this private placement have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or applicable state securities laws, and accordingly may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws. Pieris has agreed to file a registration statement with the Securities and Exchange Commission (the "SEC") registering the resale of the shares of common stock, the shares of common stock issuable upon the conversion of the Series C convertible preferred stock, and the common stock issuable upon the exercise of the warrants issued in this private placement.

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state. Any offering of the securities under the resale registration statement will only be by means of a prospectus.

On November 4, 2019 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported financial results for the third quarter ended September 30, 2019 and provided a general update on clinical and corporate developments (Press release, Deciphera Pharmaceuticals, NOV 4, 2019, View Source [SID1234550231]).

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"We believe the Breakthrough Therapy Designation we received from the FDA underscores the potential for ripretinib to transform the treatment landscape for patients with advanced GIST," said Steve Hoerter, President and Chief Executive Officer of Deciphera. "Based on the positive results from the Phase 3 INVICTUS study, we remain on track for our planned NDA submission for ripretinib in the first quarter of 2020. In addition, we provided important new clinical updates on rebastinib and DCC-3014 at the recent AACR (Free AACR Whitepaper)-NCI-EORTC meeting and we continue to progress our broad portfolio of novel product candidates all discovered here at Deciphera."

Recent Highlights and Upcoming Milestones

Ripretinib
Presented positive top-line data from the INVICTUS pivotal Phase 3 clinical study evaluating the safety and efficacy of ripretinib in fourth-line and fourth-line plus GIST patients.
FDA granted Breakthrough Therapy Designation (BTD) for ripretinib for the treatment of patients with advanced GIST who have received prior treatment with imatinib, sunitinib and regorafenib.
Company expects to submit a New Drug Application (NDA) to the FDA for ripretinib for the treatment of patients with advanced GIST who have received prior treatment with imatinib, sunitinib and regorafenib in the first quarter of 2020.
Presented updated data from the ongoing Phase 1 clinical study of ripretinib in patients with second-line through fourth-line plus GIST at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). The Company believes the updated data continue to support the ongoing INTRIGUE pivotal Phase 3 clinical study comparing ripretinib to sunitinib for the treatment of second-line GIST patients who have previously received imatinib.
Established ripretinib Expanded Access Program (EAP) for patients with locally advanced unresectable or metastatic GIST who have received treatment with prior therapies. The ripretinib EAP provides a pathway for eligible patients to gain access to this investigational medicine outside of clinical trials when no comparable or satisfactory alternative therapy option is available.
DCC-3014
Presented data from the Phase 1 dose escalation study of DCC-3014, an oral inhibitor of CSF1R, in patients with advanced solid tumors, at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). The Phase 1 data demonstrated tolerability, pharmacokinetics and biomarker mechanistic proof-of-concept in patients with advanced malignancies.
Company plans to present preliminary data from initial tenosynovial giant cell tumor (TGCT) patients at the 2019 Connective Tissue Oncology Society (CTOS) Annual Meeting being held November 13-16 in Tokyo, Japan.
Rebastinib
Presented data from the ongoing Phase 1b/2 clinical study of rebastinib, an oral TIE2 kinase inhibitor, in combination with paclitaxel at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). In Part 1 of the ongoing Phase 1b/2 study, the combination of rebastinib and paclitaxel exhibited encouraging preliminary anti-tumor activity across treatment arms and was generally well tolerated.
DCC-3116
Presented preclinical data for DCC-3116, a potential first-in-class autophagy inhibitor designed to treat mutant RAS cancers at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper). In vivo and in vitro data demonstrated DCC-3116 is a potent, selective and tight-binding inhibitor of ULK kinase, and represents a differentiated approach to autophagy inhibition and a potential first-in-class opportunity for a new therapeutic modality in mutant RAS cancers.
Recent Corporate Updates

In October 2019, Deciphera announced the appointment of Matthew L. Sherman, M.D. as Executive Vice President and Chief Medical Officer. Dr. Sherman brings over 25 years of experience as a physician-scientist in clinical drug development in oncology and hematology at leading biotechnology and pharmaceutical companies.
In the third quarter of 2019, Deciphera announced the closing of an underwritten public offering of 12,432,431 shares at a public offering price of $37.00 per share, which included the exercise in full by the underwriters of their option to purchase up to 1,621,621 shares of common stock. Total net proceeds to Deciphera were approximately $431.8 million, after deducting underwriting discounts and commissions and other offering expenses.
Third Quarter 2019 Financial Results

Cash Position: As of September 30, 2019, cash, cash equivalents and marketable securities were $634.6 million, compared to cash and cash equivalents of $293.8 million as of December 31, 2018. Deciphera expects its cash, cash equivalents and marketable securities as of September 30, 2019 will enable the Company to fund its operating expenses, capital expenditure requirements and debt service payments into 2022.
R&D Expenses: Research and development expenses for the third quarter of 2019 were $40.4 million, compared to $20.6 million for the same period in 2018. The increase was primarily due to the Company’s clinical trial costs related to the INTRIGUE pivotal Phase 3 study in second-line GIST. Personnel-related costs increased to $11.6 million primarily due to an increase in headcount and stock-based compensation expense in the research and development function. Personnel-related costs for the third quarters of 2019 and 2018 included non-cash, stock-based compensation expense of $2.0 million and $1.1 million, respectively. Facility-related and other costs included increased consultant fees of $0.4 million and increased costs incurred in connection with our early-stage drug discovery programs of $0.2 million.
G&A Expenses: General and administrative expenses for the third quarter of 2019 were $18.0 million, compared to $5.3 million for the same period in 2018. The increase was primarily due to increases in headcount and stock-based compensation expense in the Company’s general and administrative function. Non-cash, stock-based compensation was $2.7 million and $1.5 million for the third quarters of 2019 and 2018, respectively.
Net Loss: For the third quarter of 2019, Deciphera reported a net loss of $56.2 million, or $1.28 per share, compared with a net loss of $24.4 million, or $0.65 per share, for the same period in 2018.
Conference Call and Webcast

Deciphera will host a conference call and webcast to discuss this announcement today, November 4, 2019, at 4:30 PM ET. To access the live call by phone please dial (866) 930-5479 (domestic) or (409) 216-0603 (international); the conference ID is 1181263. A live audio webcast of the event may also be accessed through the "Investors" section of Deciphera’s website at www.deciphera.com. A replay of the webcast will be available for 30 days following the event.

On November 4, 2019 Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional therapeutics, reported that management will present at the upcoming Stifel 2019 Healthcare Conference taking place November 19-20, 2019 in New York, NY (Press release, Zymeworks, NOV 4, 2019, View Source [SID1234550247]).

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The Company’s presentation will be on Tuesday, November 19, 2019 at 8:00 a.m. ET.

Interested parties can access a live webcast of the presentation via a link from Zymeworks’ website at View Source, which will also host a recorded replay available afterwards.